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Shire is paying Sangamo BioSciences $13 million up front as part of a collaboration to develop therapeutics for hemophilia and other monogenic diseases, based on Sangamo’s zinc finger DNA-binding protein (ZFP) technology. The deal gives Shire exclusive worldwide rights to ZFP Therapeutics® targeting the genes for factor VII, VIII, IX, and X, as approaches for hemophilia A and B, along with three additional gene targets. The firms haven’t stated which additional targets might be included, but Sangamo says it believes its technology could have applications against disease areas of interest to Shire, including hematology and lysosomal storage disorders.

Under terms of the collaboration Sangamo will carry out all research and development activities through to the submission of IND applications and European Clinical Trial Applications for each product. Shire will reimburse its partner for internal and external research program-related costs. The U.K.-based drugs firm will be responsible for clinical development and commercialization of all products arising from the alliance, and will also make payments to Sangamo relating to research, regulatory, development, and commercial milestones, plus royalties on product sales.

“We are delighted to be partnering the first of our monogenic disease programs with Shire,” remarks Edward Lanphier, Sangamo president and CEO. “This alliance is further validation of our ZFP platform as a transformative technology for the development of novel therapeutics, which have the potential to revolutionize the treatment of a wide range of genetic diseases.”

Sangamo is focused on the development of sequence-specific ZFPs including ZFP Nucleases (ZFNs) for gene modification, and ZFP transcription factors (ZFP TFs), which can control gene expression and thus cell function. The firm says that work in a mouse model of hemophilia B has already established proof of concept that ZFN-mediated genome editing can be accomplished in vivo and and cure disease. The published studies demonstrated that a single, systemic administration of ZFNs specific for the Factor IX gene led to the production of stable and clinically meaningful levels of corrected human clotting Factor IX that are capable of normalizing clotting times.

Sangamo’s in-house R&D is focused on its SB-728 program, which is exploiting the ZFN-based technology to modify the gene encoding CCR5, the major co-receptor used by HIV to infect cells of the immune system. Lead candidate SB-728-T is an autologous ZFN-CCR5-modified T-cell product, which is being evaluated in Phase II, Phase I/II, and Phase I trials in patients with HIV and AIDS. Two Phase II studies were initiated in January.

The firm is separately carrying out preclinical work to develop a ZFN approach to modifying hematopoietic stem cells. To this end Sangamo and its collaborators at City of Hope and the University of Southern California have been been granted a $14.5 million Disease Team Research Award by the California Institute for Regenerative Medicine (CIRM).

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